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. 2023 Jan 24;8(2):e164608. doi: 10.1172/jci.insight.164608

Figure 6. scAAV9-Abt1 delivery modifies the Ighmbp2nmd/nmd phenotype.

Figure 6

(A) Illustration of the self-complementary AAV9-Abt1 vector. The chicken β-actin promoter is used to express the Abt1 cDNA along with an optimized intron within the 5′ leader sequence and a synthetic poly A site. (B–E) WT (+/+), Ighmbp2nmd/nmd, and Ighmbp2nmd/nmd mice i.c.v. injected with scAAV9-Abt1 (1.5 × 1011 or 4 × 1011 viral genomes) are evaluated for lifespan (B), weight (C), time-to-right (P7–P26) (D), and hindlimb splay (P7–P26) (E). (F–H) Representative images of WT (+/+), Ighmbp2nmd/nmd, and Ighmbp2nmd/nmd mice injected with scAAV9-Abt1 (4 ×1011 viral genomes) at P7 (F), P12 (G), and P18 (H). Statistical analyses for lifespan and weight was 2-way ANOVA with Tukey’s multiple-comparison test; data are shown as mean ± SEM. Statistical analyses for TTR and HLS was ordinary 1-way ANOVA with Tukey’s multiple-comparison test.